Treatment often based on supportive measures, including red cell and platelet transfusions. Chemotherapy agents such as azacitidine, lenalidomide, and decitabine are used for certain patients. Stem cell transplant is the only potentially curative therapy.

May have a prolonged course with anaemia and neutropenic infections, or may progress rapidly to acute myelogenous leukaemia (AML). However, most patients die of infection while in the myelodysplastic syndrome (MDS) stage of their disease.

Definition

Myelodysplastic syndrome (MDS) is a group of clonal stem cell disorders, characterised by ineffective and dysplastic haematopoiesis resulting in 1 or more cytopenias, and a varying predilection to develop acute myeloid leukaemia (AML).
[1]Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127:2391-2405.
http://www.bloodjournal.org/content/127/20/2391.long?sso-checked=true
http://www.ncbi.nlm.nih.gov/pubmed/27069254?tool=bestpractice.com
MDS is diagnosed when bone marrow demonstrates significant dysplasia, clonal cytogenetic abnormality, quantitative changes in at least one of the blood cell lines, and blasts <20%. Patients with blasts ≥20% are considered to have AML. MDS can arise primarily without any inciting event or may be related to previous treatment with either chemotherapy or radiation.

Disclosures

AKG has received research grants from Amgen, AstraZeneca, Bristol-Myers Squibb, Janssen, Merck, New Link Genetics, and Pfizer. AKG has been reimbursed for consulting work for Ariad Pharmaceuticals, Biodesix, and Pfeizer. He is also in discussion with Fresenius-Kabi on a consulting contract for clinical trial development. None of the grants or payments relate to work involving myelodysplastic syndrome.